The European Commission (EC) is expected to decide whether to approve PRX-102 (pegunigalsidase alfa) for adults with Fabry disease in early May. The therapy’s co-developers, Protalix BioTherapeutics and Chiesi Global Rare Diseases announced that PRX-102 received a positive opinion from the Committee for Medicinal Products for…
People with Fabry disease often have impairment in the nerves that help regulate heartbeat, which may serve as a prognostic measure to assess heart problems in the disease, a study reports. Mild problems with heart nerves were found in many patients in the disease’s earlier stages who didn’t have…
More than one in 10 people with Fabry disease have a form of swelling called lymphedema, a recent study reported that highlighted the importance of screening for this type of swelling, which often goes unrecognized. “Strategies to identify lymphedema in a timely manner can facilitate effective treatment and minimize…
The U.S. Food and Drug Administration (FDA) has placed a hold on the 4D-310 clinical program, an experimental gene therapy for Fabry disease being developed by 4D Molecular Therapeutics (4DMT). The hold was disclosed in a filing submitted by 4DMT to the Securities and Exchange Commission earlier this month.
One year after treatment with 4D-310, an experimental gene therapy for Fabry disease, measures of heart health have improved in the first three participants in a clinical trial, according to new data announced by the therapy’s developer, 4D Molecular Therapeutics (4DMT). “4D-310 holds promise as a potential first-in-class treatment…
A program that screened nearly 500 men with implanted pacemakers for Fabry disease found that none of them had the genetic disorder. The findings “suggest that the prevalence of [Fabry disease] in male patients requiring permanent pacing is very low and does not justify a systematic screening,” according to…
ISU303, an experimental enzyme replacement therapy for Fabry disease, showed a good safety profile and some promising signs of efficacy in a small clinical trial in Asia, according to researchers. The “results suggest that ISU303 is safe and effective and [may be an] alternative ERT for [Fabry disease],” the…
One-time treatment with ST-920 (isaralgagene civaparvovec), an experimental gene therapy being developed by Sangamo Therapeutics, continues to be generally well-tolerated among people with Fabry disease, according to new data from the Phase 1/2 STAAR clinical trial. Trial data show that the gene therapy leads to long-term increases in activity…
Implementing nationwide screening programs for Fabry disease in people with a heart condition called hypertrophic cardiomyopathy (HCM) may help improve diagnosis of the disease, a study in Czech Republic shows. In the country, the program led to the diagnosis of Fabry in four men and two women among the…
Activating a mitochondrial protein called TRAP1 can help to improve the functionality of lysosomes, the cellular structures that are defective in Fabry disease, a new study reports. The study, “Activation of mitochondrial TRAP1 stimulates mitochondria-lysosome crosstalk and correction of lysosomal dysfunction,” was published in iScience.
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